WIKIPEDIA, BEN.LAFRANCECRISPR is best known for its use in gene editing—slicing up base pairs to disable genes or correct genetic sequences. But by neutering the Cas9 nuclease typically involved in CRISPR, researchers can instead regulate the activity of a targeted gene. Using this approach, scientists have boosted levels of a protein in cells from a patient with a genetic disease called Duchenne muscular dystrophy (DMD).
People with DMD, a severe neuromuscular disorder, don’t make the protein dystrophin. But its absence can be compensated for by utrophin, a cytoskeletal protein. So Ronald Cohn of the Hospital for Sick Children in Toronto and his team used CRISPR to turn up production of utrophin.
“Remarkably, we demonstrated that several [single guide] RNAs targeting either promoter A or B upregulated utrophin amounts such that they were 1.7- to 2.7-fold or 3.8- to 6.9-fold, respectively, higher than basal amounts,” the authors wrote in their report, which will be published next month (January 7) in the American Journal of Human Genetics.
The study offers a potential therapeutic strategy to deploy CRISPR to treat this disease. “Even a modest ~1.7-fold increase in the amount of utrophin (in ...
